ESPE2021 Free Communications Bone and Mineral Metabolism (6 abstracts)
Off-label use of cinacalcet in pediatric primary hyperparathyroidism: a French multi-centre experience
Julie Bernardor 1,2,3,4 , Sacha Flammier 2,3 , Jean-Pierre Salles 5 , Mireille Castanet 6 , Laetitia Martinerie 7 , Anne Lienhardt-Roussie 8 , Cyril Amouroux 9 , Agnès Linglart 10 & Justine Bacchetta 1,2,3,11
1INSERM, UMR 1033, Faculté de Médecine Lyon Est, Université Claude Bernard Lyon1, Lyon, France.;2Centre de Référence des Maladies Rares du Calcium et du Phosphate, Filière Maladies Rares OSCAR, Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Bron, France.;3Centre de Référence des Maladies Rénales Rares, Filières Maladies Rares ORKID et ERK-Net, Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Bron, France.;4Faculté de Médecine, Université de Nice Côte d’Azur, Nice, France.;5Unite d’Endocrinologie, Maladies Osseuses, Hopital des Enfants, Toulouse University Hospital, INSERM UMR 1043 (CPTP), University of Toulouse Paul Sabatier, Toulouse, France.;6CIC INSERM U1404, Department of Pediatric, Rouen University Hospital Charles Nicolle, Rouen, France.;7Assistance Publique-Hôpitaux de Paris (AP-HP), Department of Pediatric Endocrinology and Diabetology, and Centre de Référence des Maladies Endocriniennes Rares de la Croissance, Hôpital Universitaire Robert-Debré, Paris, France.;8Paediatric Unit, Limoges University Hospital, Limoges, France.;9Paediatric Unit, Montpellier University Hospital, Montpellier, France.;10Assistance Publique-Hôpitaux de Paris (AP-HP), Hôpital Bicêtre Paris Saclay, Endocrinology and Diabetology for Children, Reference Centre for Rare Diseases of Calcium and Phosphate Metabolism, OSCAR Network, ERN BOND, Le Kremlin-Bicêtre, Paris, France.;11Faculté de Médecine Lyon Est, Université Claude Bernard Lyon 1, Lyon, France
Background: For adult patients who do not meet indications to surgery, the calcimimetic cinacalcet is approved for primary hyperparathyroidism (HPT), decreasing calcium and parathyroid hormone (PTH) levels. This drug is largely used in adults, but may be a challenge in daily practice in children, because of the risk of hypocalcemia, increased QT interval and drug interactions. A few pediatric cases have highlighted its potential interest in children with HPT; the aim here is to report the French pediatric experience in this setting.
Methods: We retrospectively analyzed data from 19 pediatric patients from 7 tertiary French centres who received cinacalcet for HPT. The results are presented as median (interquartile range).
Results: At a median age of 12.2(2.5–14.8) years, 19 patients received Cinacalcet for primary HPT (12 patients with inactive CaSR mutation, 1 patient with CD73 mutation, 1 patient with PHEX mutation, 1 patient with multiple endocrine neoplasia and 4 patients with primary HPT of unknown etiology). Cinacalcet was introduced at an estimated glomerular filtration rate (eGFR) of 123(120–131) mL/min/1.73 m2, plasma calcium of 2.98 (2.87–3.10) mmol/L, age-standardized (z score) phosphate of -2.7(-3.2;-1.9), total ALP of 214(111-225)UI/L, 25-D of 42(22-46) nmol/l, and PTH of 59(42–80) ng/L. The starting daily dose of cinacalcet was 0.7(0.6–0.9) mg/kg, with a maximum dose of 1.0(0.9–1.3) mg/kg per day. With a follow-up of 2.3(1.3–4.3) years on cinacalcet therapy, PTH remained stable at 44(35–59) ng/L (P = NS), as did eGFR, 25-D, ALP and phosphate and urinary calcium levels. In contrast, plasma calcium levels significantly decreased to 2.73(2.58–2.90) mmol/L (P = 0.03). Nephrocalcinosis was not reported but one patient displayed nephrolithiasis. Cinacalcet was withdrawn in one patient (no side effect was reported).
Conclusions: These results suggest that off-label cinacalcet in pediatric HPT controls hypercalcemia without significant side effects.
Article tools
My recent searches
My recently viewed abstracts
Authors
ESPE Abstracts
© Bioscientifica 2024 |
Privacy policy |
Cookie settings
BiosciAbstracts
Bioscientifica Abstracts is the gateway to a series of products that provide a permanent, citable record of abstracts for biomedical and life science conferences.
Find out more